ESPE Abstracts (2016) 86 P-P1-820

aDiabetology, Paediatric Endocrinology, Centre Hospitalier Universitaire d’Angers, Angers, France; bDepartment of Paediatrics, University of Chieti, Chieti, Italy; cDepartment of Paediatric Endocrinology, Hospital Arnau de Vilanova, LLeida, Spain; dNational Institute of Endocrinology, Department of Paediatric Endocrinology, Bucharest, Romania; ePediatric Endocrinology, Children’s Hospital, Charité – University Medicine Berlin, Berlin, Germany; fIpsen, Boulogne-Billancourt, France; gGenetics and Genomic Medicine Programme, UCL Institute of Child Health and Great Ormond Street Hospital for Children, London, UK

Background: The European iNCGS registry aims to collect long-term safety and effectiveness information in paediatric patients receiving NutropinAq® for growth failure.

Objective and hypotheses: To report patient baseline characteristics and exposure to NutropinAq® per country.

Method: International, multicentre, open-label, non-interventional, post-marketing-surveillance study.

Results: As of 31-Dec-2014, 3250 patients were enrolled in Germany (GE, n=1552), France (FR, n=884), Italy (IT, n=222), Spain (SP, n=387), Romania (RO, n=168), UK (n=37). Within countries, patients presented primarily with idiopathic growth hormone deficiency (IGHD), while other aetiologies were variably represented. In most countries, IGHD and Idiopathic Short Stature (ISS) were generally diagnosed at the most advanced age, Turner Syndrome (TS), Chronic Renal Insufficiency (CRI), Small for Gestational Age (SGA) at the youngest age. Height SDS at diagnosis and at commencement of NutropinAq® was lower (−3) in GE for all aetiologies (see Table 1). Except for TS, NutropinAq® was commenced at a slightly older age in FR and IT (mean (SD): 10.0 (3.6)), compared to other countries (mean (SD): 8.7 (3.6)). Time from diagnosis to treatment initiation ranged from several months (GHDs, ISS) to several years (TS, CRI, SGA). Globally, lowest initial doses were administered in GE and highest in FR (any aetiology). Dose adjustments over time were mostly carried out in GE, FR, IT. GHD patients has the same mean (SD) dose (μg/kg/day) of 30 (5) in GE/IT, and a slightly higher dose in FR: 38 (8).

Table 1. (for abstract P1-P820)
n*Mean (SD)n*Mean (SD)n*Mean (SD)n*Mean (SD)n*Mean (SD)n*Mean (SD)
Age- at first Nutropin intake (years)
GE8128.8 (3.5)899.4 (4.0)1088.2 (3.5)267.1 (5.0)1067.6 (2.9)669.7 (3.8)
FR57310.4 (3.6)859.1 (3.8)417.4 (4.1)312.0 (4.4)1248.6 (3.5)2411.0 (2.9)
IT17210.0 (3.1)28.0 (5.7)98.4 (4.6)189.9 (4.7)412.3 (2.2)411.8 (2.1)
SP2619.0 (3.7)117.4 (4.5)159.3 (3.6)107.7 (3.7)476.9 (2.9)2310.5 (3.5)
RO1238.1 (3.3)156.7 (3.3)149.4 (2.8)055.4 (2.4)112.0
Height SDS art first Nutropin intake
GE809−2.7 (0.9)89−2.8 (1.1)107−2.9 (0.8)8−2.6 (1.4)105−2.9 (1.2)66−3.0 (1.2)
FR571−2.0 (0.8)85−1.7 (1.2)41−2 0 (1.1)2−2.6 (0.3)124−2.3 (0.9)24−2.6 (0.9)
nr170−2.3 (0.9)2−3.2 (1.7)9−2.2 (0.8)5−3.8 (1.7)4−2.7 (0.5)4−2.5 (1.0)
SP258−2.1 (1.0)11−2.7 (2.0)15−2.3 (1.4)10−22 (0.9)47−24 (0.9)22−1.8 (1.4)
RO122−2.6 (1.0)15−3.1 (1.3)14−2.5 (1.0)05−3.1 (1.0)1−0.6
Initial dose of Nutropin (μg/kg/day)
GE81128 (6)8927 (6)10942 (8)837 (12)10533 (6)6628 (5)
FR57237 (7)8435 (7)4145 (9)248 (3)12541 (10)2440 (8)
ITT17231 (6)141939 (7)546 (2)431 (06)432 (1)
SP25931 (5)1130 (4)1543 (6)1045 (22)4735 (7)2337 (7)
RO12330 (6)1531 (5)1441 (9)0537 (7)113
*n available data.

Conclusion: The study shows IGHD as the main indication for NutropinAq® treatment among participating countries. The doses used were consistent with labelling recommendations for each indication. Initial doses were lower in GE; highest doses were administered in FR. Although mean age at diagnosis was as expected, treatment initiation was driven by the aetiology, often reflecting delay between diagnosis and treatment initiation.

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